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“In a single stroke with the formation and funding of Caritas, we will create what will be one of the world’s preeminent next-generation genetic medicines companies. We strongly believe that separating our business into two highly focused, stand-alone companies is the best way to unlock significant value for Amicus shareholders and to advance our medicines and technologies to people living with rare diseases around the world,” Crowley said in a statement.
Campbell, the new head of Amicus, said the company will be laser-focused on maintaining the growth of Galafold and executing on the anticipated global launch of AT-GAA.
At the same time Amicus announced the business restructuring, the company also announced that the U.S. Food and Drug Administration (FDA) had accepted its Biologics License Application for cipaglucosidase alfa and the New Drug Application (NDA) miglustat for AT-GAA, the company’s investigational two-component therapy for the treatment of Pompe disease. The FDA set a Prescription Drug User Fee Act action date of May 29, 2022, for the NDA and July 29, 2022, for the BLA.
Amicus’ AT-GAA Pompe disease treatment is a two-part approach that includes two-component therapy that consists of cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures designed to enhance uptake into cells. It is administered in conjunction with miglustat, a stabilizer of cipaglucosidase alfa. Data supporting the BLA and NDA include the Phase I/II and Phase III Propel studies and data from an open-label extension study.
Campbell, the new head of Amicus, said the company will be laser-focused on maintaining the growth of Galafold and executing on the anticipated global launch of AT-GAA.
At the same time Amicus announced the business restructuring, the company also announced that the U.S. Food and Drug Administration (FDA) had accepted its Biologics License Application for cipaglucosidase alfa and the New Drug Application (NDA) miglustat for AT-GAA, the company’s investigational two-component therapy for the treatment of Pompe disease. The FDA set a Prescription Drug User Fee Act action date of May 29, 2022, for the NDA and July 29, 2022, for the BLA.
Amicus’ AT-GAA Pompe disease treatment is a two-part approach that includes two-component therapy that consists of cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures designed to enhance uptake into cells. It is administered in conjunction with miglustat, a stabilizer of cipaglucosidase alfa. Data supporting the BLA and NDA include the Phase I/II and Phase III Propel studies and data from an open-label extension study.